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Showing 1 to 4 of 4 for “"Rna Disease"”.

  1. Strategic modification of oligonucleotides with carboxylic acids containing small molecules

    Mariscal, KevinUniversity of Ontario Institute of Technology2024

    Small interfering RNA (siRNA) are double-stranded RNA molecules used to manipulate gene regulation, by silencing gene expression. SiRNA molecules have been integrated into the therapeutic field, with several FDA-approved medications targeting liver-specific diseases. While effective, these …

    uoit Repository record

  2. Molecular Pathogenesis of Spinocerebellar Ataxia type 10

    Lim, Jung gook 1971-The University of Texas Medical Branch at Galveston2013

    … molecular pathogenesis of this unique inherited disease. In this study, we demonstrated normally transcribed ATTCT-repeats form a lengthy AUUCU pre-mRNA molecule in SCA10 cells, after being completely spliced out. We identified the specific interaction between this AUUCU RNA repeat and a …

    utmb Repository record

  3. Molecular Pathogenesis of Spinocerebellar Ataxia type 10

    Lim, Jung gookThe University of Texas Medical Branch at Galveston2013

    … expansion disorder. This heterogeneous group of disease is caused by the expanded sequences of nucleotide repeat within a target gene. Spinocerebellar ataxia type 10 (SCA10) is a unique autosomal dominant cerebellar ataxia (ADCA). The presence of seizure in addition to pancerebellar ataxia is a …

    utmb Repository record

  4. Myotonic dystrophy type one causes dysregulation of drug metabolism and fatty accumulation in the liver

    Dewald, Zachary L.University of Illinois at Urbana-Champaign2024

    Submission published under a 24 month embargo labeled 'U of I Access', the embargo will last until 2026-05-01

    uiuc Repository record